Orkambi and Cystic Fibrosis

Kirstene Hair Excerpts

Monday 19th March 2018 Westminster Hall

[Paul Masterton (East Renfrewshire) (Con)]

It is a pleasure to serve under your chairmanship, Mr Evans.

I will not go back over what Orkambi is or what it does, but it is important to remember that we are talking about only the second precision medicine for cystic fibrosis to be licensed in the United Kingdom. It has rightly been licensed for use with people over the age of 12 who have this specific form of cystic fibrosis, and both the Scottish Medicines Consortium and NICE have recognised that it is a beneficial treatment. However, neither body has recommended it for general use on the NHS, meaning that, except for on compassionate grounds in certain rare cases, many people with cystic fibrosis across the UK are simply unable to access it. Given what Orkambi can do, it is no surprise that many Scots with cystic fibrosis were disappointed by the SMC’s decision in May 2017 not to recommend it for general use on the NHS in Scotland.

Although I received a number of campaign emails ahead of the debate, it was two pieces of personal correspondence that convinced me of the need to come along today and speak. Both came from a grandparent of a young person battling cystic fibrosis: Mr Alexander from Newton Mearns whose grandson is 11, and Mrs Marshall from Eaglesham. Mrs Marshall wrote to me regarding her seven-year-old grandson:

“Through vigorous twice-daily physiotherapy and the many sports he enthusiastically takes part in, he has not lost too much of his lung capacity yet. He’s a very sociable and clever child and it is heart-breaking to know that this disease could be, at the very least, slowed down or almost eradicated by these new drugs which are not available to him. Please, too many people are suffering more than they need to and dying unnecessarily young because of the delay in approving Orkambi.”

[Kirstene Hair (Angus) (Con)]

I too am here because of the number of constituents who have been in touch but also because I lost a friend, Lucy, at the age of 19. She had so much to give. She had talent and was the most caring person you could ever meet. She dealt with it in an incredibly dignified manner, and I urge the Minister to show that same dignity in dealing with his response today.

[Paul Masterton]

My hon. Friend powerfully sums up why Vertex, the SMC, NHS Scotland, NHS England and NICE need to work together to find a way to make Orkambi available on the NHS as soon as possible, not just in Scotland but right across the United Kingdom. I understand that confidential talks are ongoing and I very much hope that there will be a positive conclusion; there is no reason to assume that an agreement cannot be reached. We have seen things happening right across Europe, and in the Netherlands a deal was struck fairly recently to allow the drug to be made available.